The FDA has granted accelerated approval for the elamipretide injection, the first treatment for Barth syndrome in patients weighing at least 30 kg.  Barth syndrome is a life threatening disease of the mitochondria. The injection works by binding the inner part of the mitochondria to enhance both its structure and function, according to a Sept. 19 news release from the agency. The FDA granted the injection accelerated approval, a regulatory pathway that allows earlier access to treatments for serious conditions that address an unmet medical need. 

The approval is based on a measure that showed improved strength for the muscle used to strengthen the leg at the knee. As a condition of the accelerated approval, the agency is requiring the drug manufacturer to conduct a post-approval randomiz

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